Atkins Robert C, Walters Mark C
Children's Hospital and Research Center at Oakland, Blood and Marrow Transplantation Program, 747 52nd Street, Oakland, CA 94609, USA.
Expert Opin Biol Ther. 2003 Dec;3(8):1215-24. doi: 10.1517/14712598.3.8.1215.
Allogeneic haematopoietic cell transplantation (HCT) is presently the only treatment which offers the possibility of a cure for patients with sickle cell disease (SCD). While approximately 84% of patients survive disease-free after human leukocyte antigen (HLA)-identical sibling donor HCT, this therapy has traditionally been reserved for patients who have suffered serious complications due to the risk of transplant-related morbidity and mortality. Typically, these sickle-related complications have included recurrent episodes of acute chest syndrome, recurrent vaso-occlusive episodes and stroke. The future of HCT for haemoglobinopathies undoubtedly will evolve as transplant-related complications are reduced and as the process of selecting patients for HCT is refined.
同种异体造血细胞移植（HCT）目前是唯一有可能治愈镰状细胞病（SCD）患者的治疗方法。虽然在接受人类白细胞抗原（HLA）匹配的同胞供体HCT后，约84%的患者可无病存活，但由于存在移植相关发病和死亡风险，这种疗法传统上仅用于出现严重并发症的患者。通常，这些与镰状细胞相关的并发症包括急性胸部综合征反复发作、反复血管闭塞发作和中风。随着移植相关并发症的减少以及HCT患者选择过程的完善，血红蛋白病的HCT未来无疑会有所发展。
